The Food and Drug Administration (FDA) has granted Orphan Drug designation to PLX-200 for the treatment of Krabbe disease.

Krabbe disease is a rare inherited disorder caused by mutations in the GALC gene that lead to a deficiency of the lysosomal enzyme, galactocerebrosidase. This results in the accumulation of galactosylsphingosine in the central and peripheral nervous systems causing demyelination and death in children within the first 2 years of life.

PLX-200 works by binding to the retinoid X receptor-α, which binds to peroxisome proliferator-activated receptor alpha (PPARα). Activation of PPARα boosts lysosome biogenesis through transcription factor EB upregulation. The investigational drug has also been shown to reduce inflammation and prevent apoptosis. 

“Because supportive care is the only available treatment option for most cases of Krabbe disease, this designation validates our scientific rationale and strongly motivates us to expedite the clinical development of PLX-200 in Krabbe disease,” said Dr Hahn-Jun Lee, MSc, PhD, President and CEO of Polaryx Therapeutics, Inc. “We are moving forward with the necessary development steps to move into the clinical study as soon as possible.”

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.


Polaryx Therapeutics receives FDA Orphan Drug designation for PLX-200 to treat Krabbe disease. News release. Polaryx Therapeutics. Accessed September 2, 2021.